hNSC-01

hNSC-01 is an investigational neural stem cell therapy derived from human embryonic stem cells, currently being developed under the leadership of Dr. Leslie Thompson at the University of California, Irvine, for the treatment of Huntington's disease. The therapy is designed to be implanted directly into the striatum, where it aims to protect existing brain cells, replace lost neurons, and rebuild impaired neural circuits. Its mechanism of action involves the release of neuroprotective proteins, specifically Brain-Derived Neurotrophic Factor (BDNF), and the reduction of mutant huntingtin protein accumulations that cause neuronal damage. Preclinical studies in mouse models have demonstrated its ability to support brain cell growth and improve behavioral abnormalities. It is currently being evaluated in a Phase 1b/2a clinical trial known as REGEN4HD.

Other names

human neural stem cellshNSCs